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Study of the Safety and Efficacy of Dichloroacetate in Glioblastoma and Other Recurrent Brain Tumours

Phase I, Open-Label, Single-Arm, Clinical and Metabolomics Study of Dichloroacetate (DCA) in Adults With Recurrent Malignant Brain Tumours.

Cancer types
Brain cancer
Trial phase


ACF funding

Why this trial

Recurrent malignant brain tumors (RMBTs) encompass two categories: 1) brain tumors originating in the brain, such as World Health Organization grade III-IV gliomas, which have recurred at least once, and 2) tumors originating elsewhere in the body that have spread to the brain at least once.

RMBTs share commonalities including increasing incidence, clinical and radiographic characteristics, pathobiology, lack of effective therapies, and a fatal outcome. Consequently, the international oncology community views phase I/II trials involving RMBTs as scientifically valid and efficient for studying promising treatments.

Despite widespread public interest and usage of dichloroacetate (DCA) as a cancer treatment, there is insufficient evidence regarding its safety and effectiveness. By supporting this trial, we aimed to contribute to the scientific understanding of DCA's potential anticancer effects, enabling informed decision-making regarding its use.

Why this intervention

The characteristics of recurrent malignant brain tumors (RMBTs) present an opportunity for the utilization of a new class of pharmacological agents known as "metabolic modulators," with dichloroacetate (DCA) being the most extensively studied clinically. Metabolic modulators like DCA exert their anti-tumor effects by inducing fundamental changes in how tumors utilize substrate fuels, such as glucose, to generate the energy needed for cancer cell proliferation and metastasis.

In both animal and human studies, DCA has demonstrated anti-tumor activity against brain tumors.

Trial design

This phase I dose-escalation trial was the first comprehensive clinical study of DCA in adults with RMBTs.

15 patients were assigned an oral DCA dose per kilogram body weight, as used in prior DCA clinical trials. A “3+3” study design was employed, which means testing 3 subjects at a given dose and escalating to the next dose if no patients experience dose limiting toxicity (DLT).

The primary outcome was to determine the safety and tolerability of DCA in this patient population. Secondary outcomes included probative studies on the metabolism of DCA in these patients and investigations of the metabolic profile of RMBTs, and the effects of DCA thereon.



Results, published here, showed that DCA can be used safely in patients with RMBT but individualisation of the dose of DCA is required to avoid side effects.



  • Erin Dunbar, MD, University of Florida, Florida, USA (Principal Investigator)
  • Peter Stacpoole, MD, University of Florida, Florida, USA (Co-investigator)


  • University of Florida, Florida, USA

Our role

Consortium building
Financial support

Why we support this trial

Benefits a population with high unmet needs

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To verify the treatments’ anticancer claim

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ACF funding

Questions about this trial?

The Anticancer Fund
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More info: NCT01111097

Author: Kristine Beckers (Trial Manager)

Last Updated: March 2024.